Abstract
Background: Metformin is a drug commonly used in individuals with type 2 diabetes, obesity, and impaired glucose tolerance. It has a strong safety profile in both children and adults. Studies utilizing the Drosophila model and knock out mouse model of fragile X syndrome (FXS) have found metformin to rescue memory, social novelty deficits, and neuroanatomical abnormalities. These studies provided preliminary evidence that metformin could be used as a targeted treatment for the cognitive and behavioral problems associated with FXS. Previously, a case series of children and adults with FXS treated with metformin demonstrated improvements in irritability, social responsiveness, language, and hyperactivity. Methods: Here, we present nine children with FXS between 2 and 7 years of age who were treated clinically with metformin and monitored for behavioral and metabolic changes. Results: Parent reports and developmental testing before and after metformin are presented. There were improvements in language development and behavior (such as lethargy and stereotypy) in most of the patients. Conclusion: These results support the need for a controlled trial of metformin in children with FXS under 7 years old whose brains are in a critical developmental window and thus may experience a greater degree of clinical benefit from metformin.
| Original language | English (US) |
|---|---|
| Article number | e956 |
| Journal | Molecular Genetics and Genomic Medicine |
| Volume | 7 |
| Issue number | 11 |
| DOIs | |
| State | Published - Nov 1 2019 |
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Keywords
- FMR1
- fragile X syndrome
- metformin
- targeted treatments
- translational medicine
ASJC Scopus subject areas
- Molecular Biology
- Genetics
- Genetics(clinical)
Cite this
Metformin treatment in young children with fragile X syndrome. / Biag, Hazel Maridith B.; Potter, Laura A.; Wilkins, Victoria; Afzal, Sumra; Rosvall, Alexis; Salcedo-Arellano, Maria Jimena; Rajaratnam, Akash; Manzano-Nunez, Ramiro; Schneider, Andrea; Tassone, Flora; Rivera, Susan M.; Hagerman, Randi J.
In: Molecular Genetics and Genomic Medicine, Vol. 7, No. 11, e956, 01.11.2019.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Metformin treatment in young children with fragile X syndrome
AU - Biag, Hazel Maridith B.
AU - Potter, Laura A.
AU - Wilkins, Victoria
AU - Afzal, Sumra
AU - Rosvall, Alexis
AU - Salcedo-Arellano, Maria Jimena
AU - Rajaratnam, Akash
AU - Manzano-Nunez, Ramiro
AU - Schneider, Andrea
AU - Tassone, Flora
AU - Rivera, Susan M.
AU - Hagerman, Randi J.
PY - 2019/11/1
Y1 - 2019/11/1
N2 - Background: Metformin is a drug commonly used in individuals with type 2 diabetes, obesity, and impaired glucose tolerance. It has a strong safety profile in both children and adults. Studies utilizing the Drosophila model and knock out mouse model of fragile X syndrome (FXS) have found metformin to rescue memory, social novelty deficits, and neuroanatomical abnormalities. These studies provided preliminary evidence that metformin could be used as a targeted treatment for the cognitive and behavioral problems associated with FXS. Previously, a case series of children and adults with FXS treated with metformin demonstrated improvements in irritability, social responsiveness, language, and hyperactivity. Methods: Here, we present nine children with FXS between 2 and 7 years of age who were treated clinically with metformin and monitored for behavioral and metabolic changes. Results: Parent reports and developmental testing before and after metformin are presented. There were improvements in language development and behavior (such as lethargy and stereotypy) in most of the patients. Conclusion: These results support the need for a controlled trial of metformin in children with FXS under 7 years old whose brains are in a critical developmental window and thus may experience a greater degree of clinical benefit from metformin.
AB - Background: Metformin is a drug commonly used in individuals with type 2 diabetes, obesity, and impaired glucose tolerance. It has a strong safety profile in both children and adults. Studies utilizing the Drosophila model and knock out mouse model of fragile X syndrome (FXS) have found metformin to rescue memory, social novelty deficits, and neuroanatomical abnormalities. These studies provided preliminary evidence that metformin could be used as a targeted treatment for the cognitive and behavioral problems associated with FXS. Previously, a case series of children and adults with FXS treated with metformin demonstrated improvements in irritability, social responsiveness, language, and hyperactivity. Methods: Here, we present nine children with FXS between 2 and 7 years of age who were treated clinically with metformin and monitored for behavioral and metabolic changes. Results: Parent reports and developmental testing before and after metformin are presented. There were improvements in language development and behavior (such as lethargy and stereotypy) in most of the patients. Conclusion: These results support the need for a controlled trial of metformin in children with FXS under 7 years old whose brains are in a critical developmental window and thus may experience a greater degree of clinical benefit from metformin.
KW - FMR1
KW - fragile X syndrome
KW - metformin
KW - targeted treatments
KW - translational medicine
UR - http://www.scopus.com/inward/record.url?scp=85073797447&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85073797447&partnerID=8YFLogxK
U2 - 10.1002/mgg3.956
DO - 10.1002/mgg3.956
M3 - Article
C2 - 31520524
AN - SCOPUS:85073797447
VL - 7
JO - Molecular genetics & genomic medicine
JF - Molecular genetics & genomic medicine
SN - 2324-9269
IS - 11
M1 - e956
ER -